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House Panel Reviews Revised 21st Century Cures Draft

May 1, 2015—On April 30, the one-year anniversary launch of the 21st Century Cures initiative, the House Energy and Commerce Subcommittee on Health held a hearing to review a revised discussion draft released the previous day [see related story]. Witnesses included Kathy Hudson, Ph.D., deputy director for Science, Outreach, and Policy at the National Institutes of Health (NIH); Janet Woodcock, M.D., director, Center for Drug Evaluation and Research at the Food and Drug Administration (FDA), and Jeff Shuren, M.D., J.D., director, Center for Devices and Radiological Health at the FDA.

Recapping the committee’s long road to the current discussion draft, Energy and Commerce Chair Fred Upton (R-Mich.), who has pioneered this effort along with Rep. Diana DeGette (D-Colo.), stated the discussion draft “incorporates the patient perspective into the regulatory process. It will increase funding for NIH. It modernizes clinical trials, including allowing for more flexible trial designs so we can customize trials based on the unique characteristics of patients most likely to benefit. 21st Century Cures will unlock the wealth of health data available so patients, researchers, and innovators can communicate and keep the cycle of cures constantly moving and improving.”

Rep. DeGette added, “After years of resource erosion and cuts, we deliver important new resources to the National Institutes of Health. We place the patient perspective at the heart of the FDA’s drug approval process. We will develop disease registries to pull information and help researchers drill into the unique and sometimes subtle patient populations. We’ll help new scientists begin their careers in research, so that our great minds tackle biomedical challenges.”

Subcommittee Chair Joe Pitts (R-Pa.) said the legislation would “invest in the basic research so critical to equipping our nation’s best and brightest with the tools they need to discover the underpinnings of disease; it would streamline the development of new therapies and technologies, which has become increasingly challenging and resource intensive; and it would foster a dynamic, continuously learning health care delivery system.”

Despite the camaraderie, Chairman Pitts noted, “Work continues on several complicated, yet critical issues, including the regulation of diagnostic tests and telemedicine.” Several members further echoed individual concerns regarding prescription drug development and affordability, drug exclusivity, interoperability, and the need to assess available resources at the FDA.

Praising the committee’s achievements, Ranking Member Pallone stated, “I am most proud to see, is $10 billion in mandatory funding for NIH over the next five years. It also includes a $1.5 billion increase in NIH discretionary authorization over the next three years. This is a real win for researchers, patients, and industry alike. I believe federal funding is the foundation of our biomedical ecosystem and is one of the most promising ways to spur economic prosperity and treatments and cures for the 21st century.”

Subcommittee Ranking Member Gene Green (D-Texas) and Reps. Jan Schakowsky (D-Ill.) and Doris Matsui (D- Calif.) echoed similar sentiments regarding the increase to NIH discretionary funding. Calling the NIH “the world's leading biomedical research” and “one of the great success stories of the federal government,” Rep. Green further urged, “Additional funding for NIH is tantamount to this effort. It is so important that the initiative includes increased funding for NIH, both through reauthorization and $10 billion over five years in mandatory funding.”

In her testimony, Dr. Hudson highlighted historic scientific and technological breakthroughs generated by NIH-supported research and spoke about the president’s Precision Medicine Initiative, which she stated would “accelerate biomedical discoveries and provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patient.”

Dr. Woodcock and Dr. Shuren jointly testified and affirmed the FDA’s commitment in working with the committee to ensure focus on “accelerating innovation, while ensuring the safety and effectiveness of products, allowing for FDA’s efficient review of drugs and medical devices,” while ensuring that new provisions will not “impede or conflict with the important ongoing work pursuant to the Prescription Drug User Fee Act (PDUFA) and the Medical Device User Fee Act (MDUFA).”

Asking Dr. Hudson results of the public health evaluation tap (Section 241 of Public Health Services Act), Chairman Upton said, “The practice of taking away 2.5 percent of NIH’s research budget through the tap must create some difficulties when planning…. [W]ould it be in the public’s best interest for the NIH to be exempt from that requirement as I understand we did in the cromnibus legislation from last year?”

Dr. Hudson responded noting the tap is still in existence but is “rerouted into NIH with a reduction in the base budget in one of our institutes, the National Institute of General Medical Sciences.” She further stated the method was “not an ideal fix for the situation.”

Regarding the proposed Innovation Fund, Rep. Joe Barton (R-Texas) said, “I support the concept. In 2006, we created the common fund and set a minimum of 1.8 percent, which is about six or seven hundred million dollars. That common fund has done great work but it’s never been increased in funding; it’s authorized up to 5 percent.”

He then asked Dr. Hudson, “Couldn’t we put this $2 billion that we’ve earmarked for the Innovation Fund and put it in to the existing common fund, because that was the whole purpose of the common fund, was to give the director the ability to move money where it would do the most good?”

Dr. Hudson replied stating the common fund has been an “amazing asset” but that the Innovation Fund more importantly “distributes funds to the institutes and centers for the innovative research.”

Rep. Joseph Kennedy III (D-Mass.) relayed prevailing concerns “from hospitals and provider communities saying that they’re losing young talented scientists to other industries or even to other countries,” and further asked Dr. Hudson to expand on the priority of young scientists in the proposed Innovation Fund.

“This is a substantial problem. We need to reach an equilibrium in the workforce pipeline, so that we can attract new investigators, and certainly young people are going to see this $2 billion mandatory funding stream as an opportunity and encouragement to stay in, and stay with the biomedical research enterprise,” said Dr. Hudson.

She further urged the necessity to fund “innovative investigator initiated research. The best ideas come from the best brains across America, and we don’t necessarily understand what those ideas are going to be until they come before us, and right now we’re only paying 18 percent of the grants that come to us. We know we’re leaving great science unfunded.”

On the same note, Rep. Kathy Castor (D-Fla.) expressed concerns that the domestic medical research is “falling behind” and “at risk of losing its competitive edge” across the globe, further urging the need to make research funding mandatory.

Reflecting on NIH budget constraints since 2003, Rep. Schakowsky recalled testimony from NIH Director Francis Collins, M.D., Ph.D., who suggested that with adequate funding, Ebola research, including a possible vaccine, may have been more developed. She then asked Dr. Hudson for other examples of what could be achieved if the NIH were adequately funded.

“One of the most devastating effects of the budget constrictions of the last several years has been the lack of appeal of careers in biomedical research in young people,” Dr. Hudson replied. “We hear repeatedly, the chronic depression of youngsters, questioning whether or not it’s worth pursuing a career in biomedical research. And that’s particularly true for M.D.’s or M.D./Ph.D.’s, who could instead be in clinical practice where there’s a more secure career trajectory, rather than research where the success rate, and we hope to see this rise, is 18 percent. So people are spending a lot of time writing grants and are not getting them funded.”